tisagenlecleucel 在 R/R B-ALL 患者中的真实世界数据：来自 CIBMTR 登记处的亚组分析

Since the first approval of tisagenlecleucel in 2017, pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) may receive this CD19-directed chimeric antigen receptor T-cell therapy.

We report real-world data from the Center for International Blood and Marrow Transplant Research (>2. 5 years of follow-up). As of 4 May 2022, 768 patients with B-ALL had received tisagenlecleucel. Patients aged ≥18 and <18 years of age (including those <3 years) were treated during first relapse (26. 6% and 26. 7% [<3 years, 44. 8%], respectively) or primary refractory disease (12. 4% and 12. 1% [<3 years, 15. 5%], respectively) with 17. 6% and 11. 6% (<3 years, 13. 8%), respectively, having high disease burden (≥50% bone marrow [BM] blasts) and 20. 2% and 20. 2% (<3 years, 13. 8%), respectively, having low disease burden (>0 to <5% BM blasts). Among patients with ≥12 months postinfusion follow-up (n = 578; median follow-up, 32.

1 months), the best overall response of complete remission/complete remission with incomplete blood count recovery was 86. 0%. The 12-month relapse-free survival (RFS) and overall survival (OS) were 61. 8% and 79. 4%, respectively, whereas the 24-month RFS and OS were 50. 3% and 63. 8%, respectively. Age (<18 years) and disease burden (<50% BM blasts) were associated with better outcomes.

Previous inotuzumab therapy and KMT2A rearrangement were associated with worse outcomes. Older patients (≥18 years) experienced a higher rate of any-grade cytokine release syndrome (CRS) associated with higher disease burden before infusion. Any-grade CRS and neurotoxicity were lower in patients aged <3 years. Extended follow-up continues to demonstrate high rates of RFS and favorable safety in this population.